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East Coast Biotech Roundup: Scholar Rock, Dicerna, Eleven, & More

Just when you thought the biotech IPO stampede was over, the holiday break came, and with it, a new batch of prospective publicly-traded life sciences companies. Those stories and more below as the roundup returns from a short vacation:

—Growth factors are well known in the biotech world, but a new Cambridge, MA-based startup named Scholar Rock wants to target them in an unusual way. Scholar Rock emerged from stealth this week and added a few well-known Boston biotech entrepreneurs like Michael Gilman and Katrine Bosley to its board of directors. I spoke with CEO Nagesh Mahanthappa about the company’s approach, which involves using what it’s calling “niche” activators and inhibitors to indirectly switch on or switch off specific growth factors.

—Watertown, MA-based Dicerna Pharmaceuticals signaled an IPO was coming when it raised a $60 million round of venture dollars in August in a round that included investors better known for their investments in public companies. Five months later, Dicerna has landed on the biotech IPO queue, announcing plans to raise up to $69 million by selling shares to public investors. Dicerna, which has a proprietary approach to silencing disease-related genes through RNA interference, has already raised $110 million since its inception. It’ll trade on the Nasdaq under the symbol “DRNA” should it pull of the IPO.

—Cambridge, MA-based Eleven Biotherapeutics may also be among the 2014 class of biotech IPOs. Just before New Year’s, the company, which is developing drugs for eye diseases like dry eye and allergic conjunctivitis, filed a prospectus with the SEC outlining a $69 million IPO of its own. Third Rock Ventures owns 41.3 percent of Eleven. Flagship Ventures (28.8 percent) and JAFCO (18.4 percent) are also significant stockholders. Eleven plans to use the cash to run a Phase 3 clinical trial of its dry eye disease drug candidate EBI-005. That trial is supposed to get underway early this year.

—Cambridge-based vaccine developer Genocea Biosciences is also one of the latest East Coast biotechs to decide to take the plunge and become a publicly-traded company. Genocea aims to raise up to $75 million from public investors through an IPO, plans that it announced just a couple days before Christmas. Genocea has raised about $81 million since its inception from investors like Polaris Partners and the Bill & Melinda Gates Foundation. It’ll use the cash to help advance vaccines it’s developing for genital herpes and pneumococcus.

—After a brutal few months, Cambridge-based Ariad Pharmaceuticals (NASDAQ: ARIA) ended 2013 on a positive note as the FDA gave it the green light to start selling its cancer drug ponatinib (Iclusig) in the U.S. once again. The news wasn’t all good, however. The FDA-approved prescribing information includes strong warning language because of safety concerns that have grown as more patients have had experience with the drug. Ariad likely won’t see the revenue numbers it had initially hoped for when the FDA first approved the drug in December 2012.

—Waltham, MA-based Tesaro (NASDAQ: TSRO) felt the wrath of investors just before the holiday break when it revealed that rolapitant, its drug for chemotherapy-induced nausea, didn’t hit its secondary goals in two Phase III clinical trials. Tesaro trumpeted the fact that the study did hit the primary mark of both studies—patients taking rolapitant didn’t vomit or need rescue medication in the “delayed” phase, or between 24 and 120 hours after starting chemotherapy—but investors weren’t impressed. Shares fell more than 20 percent.

—When Sanofi paid $20 billion for Cambridge-based Genzyme a few years, one of the supposed jewels of the buyout was a multiple sclerosis drug candidate called alemtuzumab (Lemtrada). While it’s already approved in 30 countries, that drug won’t grab a share of the U.S. market anytime soon. The FDA rejected the company’s application to sell alemtuzumab in the U.S. The agency questioned the company’s trial design and cited certain potentially serious side effects. It also recommended that the company run another clinical trial if it hopes to eventually sell the drug in the U.S. It’s a big setback for Genzyme, which had been hoping to pit alemtuzumab against Biogen Idec’s (NASDAQ: BIIB) recently-approved dimethyl fumarate (Tecfidera) in the U.S. Genzyme has said it will appeal the decision.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler